I did not describe a bureaucratic process. The FDA has researchers and clinicians on its payroll, including the director of CBER. They conduct the review to ascertain the merits of any regulatory submission. It is not filing paperwork. Just stop. You have no clue how this works.

The FDA may not be staffed by people on the cutting edge, and sponsors have to invest time in demonstrating to the agency that any new tech is justified. Those wheels can turn slowly, and they have acknowledged this. I can't speak about everyone at the agency, but I have colleagues that are ex-FDA who are at the top of their field.

An example of Windows XP just shows that software validation is not trivial. Do you think the shuttle was running state of the art code on cutting edge hardware? And why would that be?

possible for some genes, but is this case the therapy leads to the upregulation of a gammaglobin protein, normally expressed natally, to form fetal hemoglobin. Some people maintain expression of fetal hemoglobin already with no ill effects.

Its not just pain. Vasoocclusive crises can be emergent. SCD patients often have decades less life expectancy. While current SOC helps, ie hydroxyurea, the curative data from gene therapies is a huge deal, and has all but eliminated VOC.

Not sure what you actually do, but you have no idea how this works. That is exactly what the FDA does. If a sponsor wants to enter clinical trials in the US, they submit an IND to the FDA for review, which details everything about it: what it is, how it works, how its made, nonclinical safety evaluations, product testing, and representative data from multiple clinical lots demonstrating the sponsor can make it consistently and safely. The FDA will then decide if the sponsor can initiate a phase 1 trial to establish safety in a small group of patients, ie "safe to proceed". The FDA would then review and approve whether that same sponsor could proceed to a phase 2 trial, etc. Some of this is governed by federal law, ie CFR (Code of Federal Regulations) and some is covered in FDA guidance documents, and somewhat subjective on the part of the agency.

This is not true. There are hundreds of clinical trials for investigational cell and gene therapy in the US, which means they were reviewed by the FDA and given a safe to proceed to dosing patients. The FDA wants to see better therapeutic options for patients, but still has a mission to preserve patient safety.

Peter Marks, Director of CBER, was challenged on this topic recently by several in the cell and gene therapy space: our main weapons against cancer are things which themselves are known to be risk for causing cancer, so why is the agency so slow to support investigational gene therapies which may offer a much better safety profile. He conceded the FDA has been slow in this area, but looking to address this lag. There is a lot of interest in curative therapies, and "pharma" is definitely not preventing the advancement of them. It is a competitive space, and guess who wins in the compeition: company 1 with a chronic treatment or company 2 with a cure? Cost can and does feature in the equation with the payers (eg insurance companies), but generally they like cures, because the cost of health care over a lifetime is very high. And "solutions out of the lab" don't translate to human popoulations most of the time. We have cured cancer in highly inbred lab mice thousands of times, but those therapies have mostly failed to work in outbred human populations.

It is a good assumption that the data may be obtained or rules may change, but any DNA sequencing at the nucleotide level is by definition personally identifying and not anonymous. The point of the research is to attempt to correlate specific genetic variants in specific genes with clinical outcomes. The complete genomes are likely less useful here, but combinations of genetic variants could be useful. This has been attempted previously, with Decode in Iceland one of the first. The problem there was that was a limited dataset of a very specific population, ie 300K Icelanders. Getting a broader dataset of a large, outbred population could be more useful here. Testing genetic variants in highly inbred animal models often does not translate well to large, outbred human populations. This is meant to address that gap.

Really old news. MS has been doing this sort of thing for years, the old "your company is underlicensed" trick. "Would you believe you need 500 licenses?" These stories have been reported before, and IT managers have been insprired to switch to OSS as a result, although not very many.